Compassionate use of orphan drug 3,4-Diaminopyridine(DAP) in Treatment of Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP is used to decrease the muscle weakness associated with LEMS and hopefully will decrease the need for prednisone and all other therapies that were previously required to control symptoms. How long a patient will take 3,4 DAP depends upon if he/she is seeing benefits from the medication or experiencing side effects that will prevent them from continuation in the study.

Recommended maximum dosage: 20mg four times daily and if needed an additional 20 mg per day for a total of 100 mg per day. Drug must be kept refrigerated at all times.

Detailed Description:

3,4-diaminopyridine (3,4-DAP) decreases symptoms of weakness in patients with LEMS, and therefore can be used to decrease the amount of immune modulation therapy needed to provide an equivalent degree of disease control.

Eligibility

Ages Eligible for Study:

18 Years and older

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Clinical diagnosis of LEMS with or without any of the following: evidence of underlying malignancy, presence of P/Q or N-type calcium channel antibodies, electrodiagnostic evidence of a presynaptic defect of neuromuscular junction transmission.None of these laboratory findings are required for inclusion in this study.

P/Q and N type calcium channel antibodies are measured in the blood as a routine laboratory test during the course of initial diagnosis, but 10-20% of patients with LEMS do not have elevated levels of these antibodies.

Exclusion Criteria:

Hypersensitivity to any component of this medication.

History of past or current seizures.

History of asthma.

Evidence of prolonged QT syndrome. There is no absolute upper limit of normal for the QTc interval.

Family history of prolonged QTc syndrome, history of unexplained syncope, seizures or cardiac arrest.

Contacts and Locations

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To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01373333